Covadonga Paneda began her presentation by introducing Altamira Therapeutics, a company based in Basel, and their innovative x-Phore platform. This platform aims to move away from lipid-based nanoparticles and instead use peptides to achieve delivery systems that could reach areas outside the liver. The x-Phore platform includes three variants: OligoPhore for small RNAs, SemaPhore for mRNA payloads, and CycloPhore for circular RNA. These variants were used in two developmental programmes: AM-401 for KRAS-driven cancers and AM-411 for arthritis.
Paneda explained that the nanoparticles were generated by the self-assembly of peptides with any RNA, followed by a coating of albumin or hyaluronic acid. This design aims to protect the RNA, enhance stability in circulation, and improve retention in specific tissues. The platform targets tissues with inflammation and certain cancers by taking advantage of vascular leakiness and specific receptors upregulated in these conditions.
The presentation included proof of concept data showing the effectiveness of the platform in reducing tumour size and inflammation in animal models. Paneda highlighted that the nanoparticles were able to protect RNA from degradation and achieve expression for several days in vitro. The platform's design also allowed for extrahepatic delivery and selectivity, avoiding the liver and targeting other tissues.
Paneda discussed the advantages of the x-Phore platform over lipid-based nanoparticles, including better stability, easier manufacturing, and reduced immunogenicity. The platform's peptide design improved endosomal escape, ensuring that the RNA reached its target within cells. The presentation also covered the future plans for the platform, including optimising RNA sequences, scaling up the formulation process, and conducting further studies to move towards regulatory approval.
